Gene editing and gene therapy
CRISPR, base and prime editing, and the therapies that rewrite DNA to treat disease.
- WikiBase editingBase editing rewrites a single DNA letter without cutting both strands. How it works, the first patients treated, and pivotal trials in 2026.Updated
- WikiCasgevy (exa-cel)Casgevy is the first approved CRISPR medicine, an edited stem cell therapy for sickle cell disease and beta-thalassemia. Approvals, data and status.Updated
- WikiCRISPR-Cas9CRISPR-Cas9 is the RNA-guided DNA-cutting tool behind modern gene editing and the first approved CRISPR medicine. Origins, uses and limits.Updated
- WikiCRISPR TherapeuticsCRISPR Therapeutics is the Swiss-based gene-editing company behind Casgevy with Vertex, now running in vivo CRISPR trials in heart and liver disease.Updated
- WikiIntellia TherapeuticsIntellia Therapeutics develops in vivo CRISPR therapies. Lonvo-z for hereditary angioedema is under FDA review; nex-z trials resumed after a 2025 hold.Updated
- WikiLipid nanoparticle delivery for gene editingLipid nanoparticles carry CRISPR and base-editor instructions into the liver. How they work in gene editing, key trials and their limits.Updated
- WikiPersonalized (bespoke) gene editingPersonalized gene editing builds an editor for one patient's mutation. The 2025 CPS1 case, how it was made and how the FDA plans to regulate it.Updated
- WikiPrime editingPrime editing is a "search-and-replace" gene-editing method. How it works, the first human data in 2025, and the road to a first filing.Updated
- AnalysisWho can get gene-editing cures? The access and cost debateGene-editing therapies like Casgevy can cost millions and need specialist centres. The evidence on access, payment models and global reach in 2026.Updated
- AnalysisHow safe is in vivo gene editing? The 2026 debateIn vivo CRISPR therapies are nearing approval, but a 2025 trial death and AAV liver deaths raised safety questions. What the evidence shows.Updated
- ExplainerHow CRISPR gene editing works: cutting, base and prime editingHow CRISPR-Cas9 finds and cuts DNA, how base and prime editors change DNA without double-strand breaks, and why the difference matters for safety.Updated
- ExplainerHow gene therapies are delivered: ex vivo, viral vectors and LNPsHow gene editors and gene therapies reach cells: editing cells outside the body, viral vectors like AAV and lentivirus, and lipid nanoparticles.Updated